Achaogen steps up U.S. and E.U. filing plans after lead antibiotic aces pivotal trials

Analysts predict plazomicin could make peak annual sales of $340 million.

Shares in Achaogen more than doubled after the company report its lead drug—an antibiotic for serious multidrug resistant infections—met its targets in late-stage trials.

The company said it is now on track to file for approval of the next-generation aminoglycoside antibiotic in the U.S. and E.U. as a treatment for serious MDR infections, including those caused by carbapenem-resistant enterobacteriaceae (CRE), in the second-half of 2017. 

It will also step up efforts to sign up a partner for the drug outside the U.S. market and—flushed with its success—the company has announced a public offering of 5.75 million of its common stock. Price and terms have yet to be announced. 

Plazomicin matched well-established antibiotic meropenem in the EPIC registration study—meeting FDA requirements—and outperformed the comparator by EMA efficacy standards in patients with complicated urinary tract infections (cUTI) and acute pyelonephritis (AP).

Meanwhile, in a second trial called CARE in serious CRE infections, patients on plazomicin had a lower rate of mortality or serious disease-related complications compared with those on colistin, one of the few remaining antibiotics for treatment of CRE infections. 

The incidence of CRE is rising rapidly and with limited treatment options these infections are associated with high mortality rates—in some cases as high as 50%—with around 800,000 cases each year associated with bacteria that have extended spectrum beta-lactamases (ESBL), that make them resistant to a very broad range of antibiotics. 

Little surprise that the Centers for Diseases Control and Prevention (CDC) classes ESBL-positive CRE as an urgent public health threat. Carbapenems such as meropenem are now the standard of care, but cases of resistance even to these critical medicines is on the rise, according to Achaogen's chief operating officer Blake Wise. 

The firm estimates that there could 1.5 million treatment days per year for plazomicin in the U.S. and E.U. alone as a CRE therapy, he said.

Achaogen chief executive Kenneth Hillan told investors on a conference call that the trials reveal that it is possible even for a small biotech to have an "outsize impact". There is increasing concern about the emergence of MDR infections, with a UK review estimating in 2014 that by 2050 they could claim 10 million lives a year and cut global output by $100 trillion.

Analysts at William Blair said last month that plazomicin could make peak annual sales of $340 million.